A Phase II Study on Allogeneic Stem Cell Transplantation in Patients With Active Acute Leukemia

NCT ID: NCT01814488

Last Updated: 2023-03-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

101 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-07-31

Study Completion Date

2017-06-30

Brief Summary

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The experimental treatment consists in the application of a therapeutic strategy of allogeneic transplantation as a potential curative procedure in a population of patients with chemoresistant acute leukemias.

Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are based on regimens currently in standard use in the context of allogeneic transplantation.

Detailed Description

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The experimental treatment consists in the application of a therapeutic strategy of allogeneic transplantation using either a Marrow Unrelated Donor (MUD) or a Cord Blood (CB) unit or a family Haploidentical (Haplo) donor as a potential curative procedure in a population of patients with chemoresistant acute leukemias for increase the overall survival in this patients.

Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are based on regimens currently in standard use in the context of allogeneic transplantation.

The Data Safety Monitoring Board (DSMB) in collaboration with the Steering Committee (SC) will make periodic monitoring to ensure the safety of patients enrolled in to the study. In particular, DSMB will check the periodic safety reports of serious adverse events, the primary or secondary graft failure and treated related mortality (TRM) data generated by the Data Management Center. A safety report will be generated every 30 enrolled patients completed 100 days of follow-up.

The population for analysis in the trial will be the Intention to Treat (ITT) population. All patients enrolled in the study will be included in the ITT analysis.

This study will explore the feasibility, safety and efficacy of allogeneic stem cell transplantation from unrelated, cord-blood and haploidentical donor in patients with an active leukemia. Due to the lack of detailed information from literature and the absence of alternative curative options in this patient population, criteria for sample size assessment do not refer to a formal statistical power calculation. Therefore, GITMO will promote enrollment of all patients with active leukemia eligible to allogeneic SCT in all Italian centres with the aim to collect outcome variables in ITT in the widest and most representative cohort of this specific patient population.

The choice of 80 patients transplanted is based on feasibility reasons and the expected patient population with these characteristics referred to the main Italian Transplant Centres in two year. GITMO survey data on transplant activity points to an estimated accrual of 40 patients per year over a 24 months enrolment period. Criteria for defining sample size do not follow statistical power estimates in order to demonstrate difference between the alternative donor options.

Conditions

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Acute Leukemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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allogenic transplant

The experimental treatment consists in the application of a therapeutic strategy of allogeneic transplantation as a potential curative procedure in a population of patients with chemoresistant acute leukemias. Therapeutic intervention, namely the conditioning regimen as well as GVHD prophylaxis, are based on regimens currently in standard use in the context of allogeneic transplantation.

Group Type EXPERIMENTAL

allogeneic transplant

Intervention Type PROCEDURE

Allogenic transplant using either a Marrow Unrelated Donor or a Cord Blood unit or a family Haploidentical donor.

The conditioning regimen in standard use is:

Thiotepa (Tepadina) i.v. 5 mg/kg/daily (total dose 10 mg/kg) day -7 and -6;

Busulfan (Busilvex) i.v. 3,2 mg/kg/day (total dose 9,6 mg/kg) as a single daily dose day -5, -4, -3;

Fludarabine i.v. 50 mg/m2 (total dose150 mg/m2) day -5, -4, -3.

Primary antifungal prophylaxis is Micafungin 50 mg/die i.v. (1 mg/kg if \<40 kg) day 0 to engraftment. After engraftment continue antifungal prophylaxis according to local practice.

Interventions

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allogeneic transplant

Allogenic transplant using either a Marrow Unrelated Donor or a Cord Blood unit or a family Haploidentical donor.

The conditioning regimen in standard use is:

Thiotepa (Tepadina) i.v. 5 mg/kg/daily (total dose 10 mg/kg) day -7 and -6;

Busulfan (Busilvex) i.v. 3,2 mg/kg/day (total dose 9,6 mg/kg) as a single daily dose day -5, -4, -3;

Fludarabine i.v. 50 mg/m2 (total dose150 mg/m2) day -5, -4, -3.

Primary antifungal prophylaxis is Micafungin 50 mg/die i.v. (1 mg/kg if \<40 kg) day 0 to engraftment. After engraftment continue antifungal prophylaxis according to local practice.

Intervention Type PROCEDURE

Other Intervention Names

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Marrow Unrelated or Cord Blood or family Haploidentical donor

Eligibility Criteria

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Inclusion Criteria

Diagnosis of Primary induction Failure or chemoresistant relapse in Acute Myeloid Leukemia (AML) or Acute Lymphoblastic Leukemia (ALL) patients

Activation of an alternative donor search by the Italian Bone Marrow Donor Registry (IBMDR)

Age \>=18\<=70

Unavailability of a HLA-matched related donor (MRD)

Performance status: ECOG\<=3

Written and signed informed consent

Life expectancy not severely limited by concomitant illness.

Exclusion Criteria

Previous allogeneic transplant (autologous transplant is accepted)

Positive pregnancy test

Any active, uncontrolled infection.

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Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Gruppo Italiano Trapianto di Midollo Osseo

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Fabio Ciceri, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale San Raffaele

Locations

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Azienda Ospedaliera SS Antonio e Biagio

Alessandria, , Italy

Site Status

Policlinico

Bari, , Italy

Site Status

Divisione di Ematologia - Ospedali Papa Giovanni XXIII

Bergamo, , Italy

Site Status

Ospedale San Orsola

Bologna, , Italy

Site Status

Ospedale Regionale Generale- Divisione Ematologia

Bolzano, , Italy

Site Status

Ospedale Binaghi

Cagliari, , Italy

Site Status

Ospedale Oncologico Businco

Cagliari, , Italy

Site Status

Ospedale Ferrarotto - Ematologia

Catania, , Italy

Site Status

S.C. Ematologia - Azienda Ospedaliera S. Croce e Carle

Cuneo, , Italy

Site Status

Cattedra di Ematologia - Azienda Ospedaliera di Careggi

Florence, , Italy

Site Status

Ospedale Policlinico San Martino - IST

Genova, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Ospedale Niguarda Ca' Grande

Milan, , Italy

Site Status

Ospedale San Raffaele

Milan, , Italy

Site Status

Divisione Ematologia - Azienda Ospedaliera Universitaria - Policlinico -

Modena, , Italy

Site Status

Cattedra di Medicina Interna ed Ematologia - Ospedale S. Gerardo de' i Tintori - Università degli Studi di Milano

Monza, , Italy

Site Status

AO Ospedali Riuniti Villa Sofia - Cervello

Palermo, , Italy

Site Status

Dipartimento Oncologico La Maddalena

Palermo, , Italy

Site Status

Fondazione IRCCS San Matteo

Pavia, , Italy

Site Status

Dip. di Ematologia - Unità di Terapia Intensiva Ematologica per il Trapianto Emopoietico - Ospedale Civile di Pescara

Pescara, , Italy

Site Status

Ospedale G. Da Saliceto di Piacenza

Piacenza, , Italy

Site Status

Cattedra di Ematologia - Policlinico

Roma, , Italy

Site Status

Policlinico A. Gemelli

Roma, , Italy

Site Status

Policlinico Universitario Tor Vergata

Roma, , Italy

Site Status

U.O. di Ematologia e Trapianti di Midollo Osseo - Azienda Osp. S. Camillo-Forlanini / Padiglione Morgagni

Roma, , Italy

Site Status

Ematologia e Centro Trapianti Midollo Osseo - Ospedale IRCCS Casa Sollievo della Sofferenza

San Giovanni Rotondo, , Italy

Site Status

Ospedale San Giuseppe Moscato

Taranto, , Italy

Site Status

Azienda ospedaliera Città della Salute e della Scienza

Torino, , Italy

Site Status

Centro Trapianti Metropolitano

Torino, , Italy

Site Status

A.O. Santa Maria della Misericordia

Udine, , Italy

Site Status

Countries

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Italy

Other Identifiers

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2012-004008-37 Gandalf-01

Identifier Type: -

Identifier Source: org_study_id

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